Hereditary Spastic Paraplegia Type 52

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Review clinical trials related to Hereditary Spastic Paraplegia Type 52. Use filters to narrow results by trial status, phase, treatment, biological sex and sponsor.

Condition / disease
Location
Status: Not yet recruiting

Safety and Efficacy of AAV9/AP4B1 (BFB-101) For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47)

Safety and Efficacy of AAV9/AP4B1 For Patients with AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47): A Phase 1/2 Single-Center, Open-Label Study of Stereotactic Intra-cisterna Magna Administration. The goal of this clinical trial is to evaluate whether a gene therapy can safely treat children with SPG47, a rare genetic condition that causes progressive spasticity and developmental delays. The main questions it aims to answer are: * Is the gene therapy safe and well tolerated? * Does the gene therapy improve motor function and developmental outcomes? Participants will: * Undergo screening assessments to confirm eligibility * Receive a single dose of the gene therapy vector * Attend follow-up visits for safety monitoring and developmental assessments over the course of five years

Participants needed: 5
Trial details
Phase: Phase 1, Phase 2Age: 12-60Biological sex: AllType: InterventionalSponsor: BlackfinBio LtdUpdated: Apr 28, 2025Locations: 1
Eligibility criteria

Male and females between the ages of 12 months - 5 years at the time of treatmen... [+8]

Inability to participate in the clinical evaluation as determined by the princip... [+19]